For gene therapy to realize its clinical potential, there mustbe efficient and safe strategies of delivering therapeutic genesto somatic cells in vivo. Perhaps this problem simply representsa special case of drug delivery in which the therapeutic geneconstitutes the drug. But the relevance of traditional drugdevelopment to technological developments in gene therapy isquestionable. Recombinant genes that can independently expressa therapeutic RNA or protein are extraordinarily large and polarmolecules — 10,000 times larger than traditional pharmaceuticalagents. Moreover, the administration of genes as therapy requiresvehicles —so-called vectors — that encapsulate thegene and . . . [Full Text of this Article]
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From the Institute for Human Gene Therapy and the Department of Molecular and Cellular Engineering, University of Pennsylvania, and the Wistar Institute -- both in Philadelphia.
Address reprint requests to Dr. Wilson at the Institute for Human Gene Therapy, 204 Wistar Institute, 3601 Spruce St., Philadelphia, PA 19104-4268.
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